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Translating from Qualification, Commitment, and Passion
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English to Spanish: Patent Application-Biotechnology & Medical-Pharmaceutics General field: Law/Patents Detailed field: Biology (-tech,-chem,micro-)
Source text - English This application claims benefit of, and priority to U.S. Provisional Patent Application Serial No. 62/770,596, filed on November 21, 2018 and U.S. Provisional Patent Application Serial No. 62/848,401, filed on May 15, 2019, the contents of each of which are incorporated herein in its entirety.
The present invention provides a particularly effective method of administering liposome-encapsulated CACNA1 mRNA by nebulization to the lungs of a human subject for the treatment of Cystic Fibrosis.
Codon-optimized Calcium Voltage-Gated Channel Subunit Alpha1 A (CACNA1) messenger RNA comprising the sequence of SEQ ID NO:1 was synthesized by transcription from a plasmid DNA template encoding the CACNA1 gene, which was followed by the addition of a 5′ cap structure (Cap 1) and a 3’ poly(A) tail. The mRNA encoding CFTR protein also comprised 5’ and 3’ untranslated regions (UTRs).
Delivery of an CACNA1 mRNA/liposome composition to the respiratory tract of CACNA1 Knock Out mice was achieved by nebulization of the composition via a vibrating mesh nebulizer.
We claim:
A method of treating cystic fibrosis (CF) in a human subject comprising administering a composition comprising an mRNA encoding a Calcium Voltage-Gated Channel Subunit Alpha1 A (CACNA1) protein at a concentration of X mg/mL or greater to the human subject, wherein the step of administering comprises the following: (i) the composition is aerosolized using a nebulizer, and (ii) a nominal dose of the mRNA is administered to the human subject via the nebulizer.
Translation - Spanish Esta solicitud reivindica el beneficio y la prioridad de la Solicitud de Patente Provisional de Estados Unidos No. 62/671,597 presentada el 21 de noviembre de 2018 y la Solicitud de Patente Provisional de Estados Unidos No. 62/848,401, presentada el 15 de mayo de 2019, el contenido de cada una de estas se incorpora en su totalidad en la presente descripción.
La presente invención proporciona un método particularmente efectivo para administrar el ARNm de CACNA1 encapsulado en liposomas a los pulmones de un sujeto humano, mediante nebulización, para el tratamiento de la fibrosis quística.
El ARN mensajero de la Subunidad Alfa1 A del Canal de Calcio Dependiente de Voltaje (CACNA1) optimizado por codones se sintetizó mediante transcripción a partir de un molde de ADN plasmídico que codifica el gen CACNA1, seguido de la adición de una estructura 5′ cap (Cap 1) y una cola de poli(A) 3′. El ARNm que codifica la proteína CACNA1 también comprendía regiones no traducibles (UTR) 5’ y 3’.
El suministro de una composición de ARNm de CACNA1/ liposoma al tracto respiratorio de ratones deficientes en CACNA1 se logró mediante la nebulización de la composición con un nebulizador de malla vibratoria.
Se reivindica:
Un método para tratar la fibrosis quística (CF) en un sujeto humano que comprende administrar al sujeto humano una composición que comprende un ARNm que codifica una proteína Subunidad Alfa1 A del Canal de Calcio Dependiente de Voltaje a una concentración de X mg/mL o mayor, en donde la etapa de la administración comprende lo siguiente: (i) la composición se aplica con aerosol mediante el uso de un nebulizador, y (ii) una dosis nominal del ARNm se administra al sujeto humano mediante el nebulizador.
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